Comparison of Ideal vs. Actual Weight Base Factor Dosing

Hemophilia A Clinical Trial

Official title:

Comparison of Ideal vs. Actual Weight Base Factor Dosing

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03286153
• Other study ID #: BDC Ideal Body Weight Dosing
• Status: Recruiting
• Phase: N/A
• First received: August 9, 2017
• Last updated: September 13, 2017
• Start date: January 2017
• Est. completion date: December 2018

Study information

• Verified date: September 2017
• Source: Bloodworks (Puget Sound Blood Center)
• Contact: Heidi Thielmann, PhD
• Phone: 206-689-6234
• Email: hthielmann[@]bloodworksnw.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a randomized, prospective, multicenter study to examine whether or not the current recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired result of bleeding prevention and cessation. This study also examines ways to prevent delivering excessive factor by using a patient's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in protective factor levels.

Description:

This is a randomized, prospective, multicenter, open-label, crossover study to examine whether or not the current recommended factor dosing strategy, i.e., dosing by actual body weight in overweight and obese patients, may deliver more clotting factor than necessary to cause bleeding to stop in participants with Hemophilia A who use Factor VIII (FVIII). This study also examines ways to prevent delivering too much factor by using a participant's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in hemostatic factor levels.

The study will be conducted at the Washington Center for Bleeding Disorders (WCBD) at Bloodworks Northwest, Oregon Health & Science University (OHSU), Seattle Children's Hospital (SCH), and Providence Sacred Heart Children's Hospital (SH). Cumulatively across the four sites, up to 20 participants will be enrolled. Randomization will be performed centrally at WCBD.

Participants will provide their own factor. Prior to the first study-related dose, participants will stop taking any FVIII products for either 48 hours if currently using a short-acting FVIII product or 72 hours for a long acting FVIII product. Factor levels will be measured immediately before and at multiple points after two different factor doses. Subjects will be randomized to start their dosage based either on actual body weight or ideal body weight first and then crossover to receive dosage based on the other category.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: December 2018
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Hemophilia A

• Able and willing to comply with pharmacokinetic testing schedule

• Either overweight or obese BMI using CDC definitions by age

Exclusion Criteria:

• Inhibitor of > 0.6 BU twice in the past, or documented abnormal recovery of less than 66% (of expected) in the past

• Known other bleeding disorder

• Known other prolongation in aPTT (lupus anticoagulant, FXII deficiency)

• Female

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Other:
• Ideal Body Weight First
Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on ideal body weight. For participants age 12-19, ideal weight is calculated using the McLaren method. For participants age 20 and over, ideal weight is calculated using the following equation: [50kg + (2.3kg*every inch over 5 feet)].
• Actual Body Weight First
Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight.

Locations

Country	Name	City	State
United States	Oregon Health & Science University	Portland	Oregon
United States	Seattle Children's Hospital	Seattle	Washington
United States	Washington Center for Bleeding Disorders at Bloodworks Northwest	Seattle	Washington
United States	Providence Sacred Heart Children's Hospital	Spokane	Washington

Sponsors (4)

Lead Sponsor	Collaborator
Bloodworks (Puget Sound Blood Center)	Oregon Health and Science University, Providence Health & Services, Seattle Children's Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Recovery	Compare the recovery with FVIII between doses calculated on actual body weight versus ideal body weight in subjects with Hemophilia A	Change from baseline at up to two months
Primary	Underdosing	Determine the likelihood of underdosing when using ideal body weight	Change from baseline at up to two months
Primary	Overdosing	Determine the likelihood of overdosing when using actual body weight	Change from baseline at up to two months
Secondary	Effect of half-life	Determine the effect on half-life of these dosing strategies	Change from baseline at up to two months
Secondary	Effect on hemophilia severity	Determine the effect of pharmacokinetic differences on hemophilia severity	Change from baseline at 20-40 minutes, 5-7 hours, 20-26 hours, and 44-50 hours for both half-life and extended half-life and also at 69-75 hours, and 93-99 hours for extended half-life
Secondary	Regular half-life vs. extended half-life Regular half-life vs. extended half-life	Determine differences in participants receiving regular half-life versus extended half-life products	Change from baseline at up to two months
Secondary	Overweight vs. obese	Determine the differences, if any, between overweight and obese participants	Change from baseline at up to two months